推进儿科精准医学;过去、现在和未来

Cambridge prisms, Precision medicine Pub Date : 2023-01-10 eCollection Date: 2023-01-01 DOI:10.1017/pcm.2022.14
Abdelbaset Elzagallaai, Charlotte Barker, Tamorah Lewis, Ronald Cohn, Michael Rieder
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引用次数: 0

摘要

精准医学是一种考虑到每个人的遗传、环境、治疗和生活方式等个体差异的疾病治疗和预防方法。这种全面的治疗方法旨在提高药物的疗效和安全性,不仅适用于整个医疗系统,也适用于个体患者。虽然在确定儿童用药剂量时考虑了体重,在一定程度上也考虑了胎龄,但从历史上看,包括遗传变异在内的其他因素并没有被纳入治疗决策的考虑范围。随着我们对本体和遗传学在决定药物疗效和安全性方面的作用的认识不断加深,这些见解为将精准医学原则应用于婴幼儿和青少年的治疗提供了新的机遇。这些机会最有可能首先在特定的儿童亚群中实现。虽然在儿童中成功实施精准医疗面临着许多挑战,包括需要确保精准医疗能够提高而不是降低儿童医疗保健的公平性,但同时也存在着更多的机遇。为实现安全有效的药物治疗这一共同目标,需要通过研究、宣传、规划和团队合作来推动精准医学在儿童中的应用。
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Advancing Precision Medicine in Paediatrics: Past, present and future.

Precision Medicine is an approach to disease treatment and prevention taking into account individual genetic, environmental, therapeutic and lifestyle variability for each person. This holistic approach to therapeutics is intended to enhance drug efficacy and safety not only across healthcare systems but for individual patients. While weight and to some extent gestational age have been considered in determining drug dosing in children, historically other factors including genetic variability have not been factored into therapeutic decision making. As our knowledge of the role of ontogeny and genetics in determining drug efficacy and safety has expanded, these insights have provided new opportunities to apply principles of Precision Medicine to the care of infants, children and youth. These opportunities are most likely to be achieved first in select sub-groups of children. While there are many challenges to the successful implementation of Precision Medicine in children including the need to ensure that Precision Medicine enhances rather than reduces equity in children's health care rather, there are many more opportunities. Research, advocacy, planning and teamwork are required to move Precision Medicine forward in children in pursuit of the common goal of safe and effective drug therapy.

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